NCT05402345

A Study of GlcNAc on Tear Production in NGLY1-CDDG

A Phase II Randomized, Multicenter, Double-Blind, Placebo-Controlled Study Evaluating Effect Of GlcNAc On Tear Production In Individuals With NGLY1-CDDG

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Eva Morava-Kozicz · Academic / Other
• Sex: All
• Age: 1 Year – 60 Years
• Healthy volunteers: Not accepted

Summary

In patients with NGLY1-CDDG, the disorder can lead to eye damage due to not being able to produce enough tears. This study is being done to see if the dietary supplement, GlcNAc, improves tear production in patients with NGLY1-CDDG.

Detailed description

This study is a multicenter randomized, double-blind, placebo-controlled trial of GlcNAc supplementation for improvement of tear production in NGLY1-CDDG. Clinical history and screening data will be reviewed to determine subject eligibility. If a subject is already on GlcNAc, a washout period of 1 month will be required prior to consent and randomization. Interested subjects who have a molecularly confirmed diagnosis of NGLY1-CDDG will be consented. Baseline data will be collected prior to randomization at treatment initiation. Subjects will then be randomized to placebo or GlcNAc. They will be administered weight-dependent doses of GlcNAc or an equivalent volume of placebo enterally for 6 weeks, followed by open label weight-dependent doses of GlcNAc for 6 weeks. A visit for evaluation and collection of lab samples will be conducted at 6 and 12 weeks.

Conditions

• NGLY1 Deficiency

Interventions

Type	Name	Description
DRUG	GlcNAc-GlcN	GlcNAc powder - weight-dependent dose
OTHER	Placebo	Placebo xylose

Timeline

• Start date: 2025-01-24
• Primary completion: 2026-12-01
• Completion: 2026-12-01
• First posted: 2022-06-02
• Last updated: 2025-07-08

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study